ADPKD Trials Update 2024/2025
Written on . Posted in News and Events.
Tolvaptan is the only approved drug therapy for adults with ADPKD at CKD stages 2-4. New trials are underway to assess children and ARPKD.
Many trials against several different targets have failed in recent years, including lixivaptan, lanreotide, pasireotide (target AVPR2); Venglustat (target lipid); everolimus, sirolimus (target mTOR); bardoxolone (target TF, NRF-2); simvastatin, etanercept (target metabolic pathways).
Ongoing and Planned Trials
Active Phase 3:
- Tolvaptan in children and ARPKD (AVPR2) - Otsuka
- Metformin "IMPEDE" UK (metabolic) - National Institute Health Research (NIHR), UK
Phase 2/3:
- Saracatinib, Bosutinib (Src TKI) - Commercial
- Tesevatinib ARPKD (Erb TKI) - Commercial
- Xorlo oxypurinol (xanthine oxidase) - Commercial
Phase 1:
- RGLS8429 Mir-17 - Commercial
- RNA activation - Commercial
- VX-407 Protein misfolding - Commercial
- PDE activator - Commercial
- PXL770 AMPK orphan drug status - Commercial
- Monoclonal Ab - Commercial